Drug Information Q&A Q50. How are iPS cells used in the development of new drugs?
Answer
iPS cells, which were created in 2006, are expected to be used to elucidate the causes of diseases, develop new drugs, and for regenerative medicine such as cell transplantation therapy.
Explanation
iPS cells are short for "induced pluripotent stem cells," and are created by injecting four genes into human skin and other cells and culturing them for several weeks. These cells are called pluripotent stem cells because they have the ability to "differentiate into various tissues and organs" and "proliferate almost indefinitely.
In addition to applications in regenerative medicine, there are attempts to use iPS cells to create new drugs.
In the development of a new drug, the effects and side effects are verified in animals, and then clinical trials are conducted to confirm the effects and side effects in humans. However, there are many similarities and differences between humans and animals, and even if a drug is successful in animal studies, its development may be halted at the human clinical trial stage. This will enable safer and more efficient drug development.
It is also hoped that iPS cells can be used to develop drugs for intractable diseases such as Alzheimer's disease, Parkinson's disease, ALS (amyotrophic lateral sclerosis), and muscular dystrophy.
By creating and differentiating iPS cells from the body cells of patients with difficult-to-treat diseases, it is possible to create disease-specific cells. By studying how the condition and functions of these disease-specific cells change, it may be possible to elucidate the causes of previously unknown diseases and to develop therapeutic drugs.
In May 2013, the Japan Pharmaceutical Manufacturers Association launched the "Consortium for Safety Assessment of Human iPS Cells" with the aim of strengthening Japan's drug discovery technology and infrastructure by quickly linking Japan-originated iPS cells to practical applications. Numerous companies and institutions, including pharmaceutical companies, contract research organizations, the National Institute of Biomedical Innovation, the National Institute of Biomedical Innovation, and the Kyoto University iPS Cell Research Institute, are participating in this consortium.
However, iPS cell research has just begun. Many scientific, medical, ethical, and other issues must be overcome before iPS cell research can be put to practical use.
Chart/Column
50｜Establishment of iPS cells
Source: Adapted from the website of the "iPS Cell Research Institute," Kyoto University.
iPS Cell Research Institute, Kyoto University
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